The Financial Times has published today a long review about the orphan drugs market, which has become more and more important for the big pharma over the last few years, as proven by the recent M&A deals. Sanofi some weeks ago paid almost $12bn for Bioverativ, a biotech focusing on research for therapies for rare blood diseases . Shire acquired Baxalta in 2016 for $32bn, seeking to become a leader in the rare diseases treatments business.
Consultancy EvaluatePharma has estimated that the business will increase from $127bn (2017) to $217bn by 2022. The top orphan drugs include Teva’s Copaxone, with revenues over $3.8bn, Alexion’s Soliris ($3.2bn), Shire’s Advate ($2.3bn), Biogen’s Avonex ($2.2bn) and Merck KGaA’s Rebif ($1.8bn). Significantly, this is a narrow market, due to the small number of global cases, which forces drugmakers to set very high prices. The two most significant cases are perhaps GSK’s Strimvelis, a gene therapy for insufficient adenosine deaminase levels, whose price has been set to $700,000 per treatment, and Biomarin’s Brineura, sold at GBP500,000.